Fujisawa

Researchers are investigating the safety and effectiveness of oral rilzabrutinib compared to a placebo in adults aged 18 and older diagnosed with primary warm autoimmune hemolytic anemia (wAIHA). This Phase 3, double-blind, parallel-group study aims to assess how well rilzabrutinib can achieve a durable hemoglobin response in about 90 male and female participants with this condition, who have had it for at least six months and have specific treatment histories related to corticosteroids. After a 4-week screening period, participants are randomly assigned in a 2:1 ratio to receive either oral rilzabrutinib or placebo for up to 24 weeks during the primary analysis period. Following this, all participants who complete this phase enter an open-label period where they receive rilzabrutinib for 28 weeks. Those who show an increase in hemoglobin during the last 8 weeks of this open-label phase may continue into a long-term extension lasting until the last participant has completed 52 weeks in this phase. During the study, participants will undergo regular assessments including measuring hemoglobin levels to determine response, safety evaluations, and pharmacokinetic and pharmacodynamic monitoring. The main outcome is the proportion of participants achieving a durable hemoglobin response defined as an increase of at least 2 g/dL from baseline on two-thirds of visits between Week 12 and Week 24. Safety follow-up will continue for 2 weeks after treatment completion or discontinuation, ensuring close monitoring throughout the study duration.

The purpose of the study is to evaluate whether ibuzatrelvir is effective and safe in adults and adolescents with COVID-19 who do not need to be in the hospital but who are at high risk for progression to severe disease. Eligible participants will be randomly assigned (by chance) to receive ibuzatrelvir or matching placebo orally for 5 days. Co-administration of locally available standard of care is allowed. The total duration of the study is around 6 months.

Researchers are evaluating the effect of muvalaplin on reducing cardiovascular risk in adults with elevated lipoprotein(a) levels who either have atherosclerotic cardiovascular disease or are at risk for a heart attack or stroke. This Phase 3, randomized, double-blind, placebo-controlled study focuses on adults with high Lp(a) levels and prior or potential cardiovascular events. The study aims to assess the time to the first major adverse cardiovascular event over about 5.25 years. Participants will be randomly assigned to receive either muvalaplin or a placebo, both administered orally. The study includes individuals with Lp(a) levels of at least 175 nanomoles per liter who have had a prior cardiovascular event within 10 years or are at risk for a first event due to conditions such as coronary artery disease, carotid stenosis, peripheral artery disease, high coronary artery calcium score, reduced kidney function with diabetes, or other high-risk factors. The treatment period lasts through the study duration, with close monitoring. During the study, participants will be regularly evaluated to track the occurrence of major adverse cardiovascular events, including heart attacks and strokes. Safety assessments will monitor blood pressure, kidney function, and heart failure status among other health indicators. The primary outcome measures the time to the first major cardiovascular event from baseline up to the end of the study, which spans approximately 5.25 years.